LOS ANGELES--(BUSINESS WIRE)--NantBioScience, subsidiary of NantWorks, LLC, a company focused on the discovery and development of innovative treatments for diseases with high unmet medical needs, today announced a strategic collaboration with Celgene to advance bold research programs to benefit cancer patients in need of new therapeutic solutions. As part of the transaction, Celgene will pay $75 million in funding to NantBioScience as an upfront option fee and equity investment.

Building on the nab® (nanoparticle albumin-bound) technology platform, NantBioScience will create a pipeline of nab®-based molecules. As part of the collaboration, Celgene will license two nab® product candidates to NantBioScience, both of which had previously received Investigational New Drug (IND) approval. The first product candidate (NTB-011) is a nanoparticle albumin-bound formulation of a novel colchicine dimer with cytotoxic and vascular disrupting properties. The second product candidate (NTB-010) is a nanoparticle albumin-bound formulation of the geldanomycin analogue, 17-AAG, a potent HSP90 inhibitor, which is planned to be studied in patients with a variety of hematological and solid tumors. Phase I clinical trials for these product candidates are being planned for initiation in 2014 - 2015.

The objective of NantBioScience is to innovate drug development by testing molecularly targeted drugs, based on the molecular profile of the patient's tumor, independent of the cancer's anatomical type. With capabilities of next generation sequencing and targeted proteomics, each cancer may now be viewed as a series of rare diseases. These comprehensive “omic” analytic tools and "big data" generated from supercomputing have been previously untapped on the scale now available in the field of drug development. NantBioScience and NantWorks are uniquely positioned to develop molecularly designed drugs in this era of genomics and proteomics, by identifying patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive “omic” analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects.

"We intend to make obsolete the standard method of clinical trial design of 'trial and error' and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. We also intend to make obsolete the common understanding that cancer treatments, developed under the age old dogma of 'maximum tolerated dose,' may work but only by wreaking terrible side effects, bringing patients to the brink of death,” said NantWorks founder, Dr. Patrick Soon-Shiong. "Celgene has been a strong steward for Abraxane which is now approved for metastatic breast, lung and pancreatic cancer. This new partnership will enable us to aggressively advance our drug pipeline and put us one step closer to developing – and then delivering – molecular designed cancer treatments for patients to receive the right care at the right time."

"We have invested over $100 Million in the pursuit of this platform to date and are very excited to have Celgene as our partner in this pursuit,” he said.

"Celgene is excited to team up again with Dr. Patrick Soon-Shiong, the creator of Abraxane® and the founder of the nab® technology platform. We are committed to his vision of molecularly driven personalized medicine and to collaborating with NantBioScience at the forefront of this era where genomic- and proteomic- based solutions may provide the path to a cure for cancers," said Bob Hugin, Chairman and Chief Executive Officer of Celgene Corporation.

In addition to the two nab® product candidates, NantBioScience has a broad R&D program to discover new compounds that are specifically targeted at tumor signaling pathways in patients with specific genetic mutations. A novel inhibitor of oncogenic KRAS is showing promise in early development studies and will advance into a program of IND enabling studies in 2014. Normally a proto-oncogene, KRAS, when mutated to an oncogene, is transformed into the driver of tumorigenesis in pancreatic cancer amongst many other cancers and molecular profiles.

NantBioScience’s pursuit of targeted therapies includes the discovery and development of drugs that remediate the activities of the often mutated tumor suppressor, p53. Because the loss of p53 functionality is a driver for the development of over 50% of all cancers and because p53 is responsible for maintaining the integrity of the cellular genome, pharmaceuticals targeting cells harboring p53 mutations are highly coveted. NantBioScience plans to initiate IND-enabling studies on its lead p53 remediating compound, for which NantBioScience is targeting a first-in-man study in 2015.

NantBioScience’s pipeline also contains novel potent multi-kinase inhibitors. These compounds are entering into a program of IND-enabling studies, with first-in-man studies planned for 2016. NantBioScience’s kinase inhibitor program is further buoyed by its library of >4,000 multi-kinase inhibitors which are currently under investigation for a variety of indications and molecular profiles.

As part of the collaboration, Celgene will receive an option to license a certain number of product candidates developed by NantBioScience, including the two nab® product candidates to be licensed to NantBioScience. The options may be exercised by Celgene through completion of Phase I clinical studies.

The transaction is subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and is expected to close in the first quarter of 2014.

About NantBioScience

NantBioScience, a wholly-owned subsidiary of NantWorks, LLC, is a biopharmaceutical company focused on the discovery of innovative treatments by developing molecularly targeted drugs, based on the molecular profile of the patient's tumor, independent of the cancer's anatomical type. Dr. Patrick Soon-Shiong, the creator of Abraxane® and the founder of the nab® technology platform, established NantBioScience to develop a pipeline of nab®-based molecules, and inhibitors of proteins which drive tumor growth. NantBioScience's mission is to make obsolete the standard method of clinical trial design of "trial and error" and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. The Company will tap into comprehensive “omic” analytic tools and "big data" generated from supercomputing to develop molecularly designed drugs in this era of genomics and proteomics and identify patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive “omic” analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects. For more information please visit www.nanthealth.com.